iCo TherapeuticsTSX-V: ICO
604-602-9414
www.icotherapeutics.com
Forward Looking Statements
Certain of the statements contained in this presentation
are forward-looking statements which involve known and
unknown risks, uncertainties and other factors which may
cause the actual results, performance or achievements of the
Company, or industry results, to be materially different from
any future results, performance or achievements expressed
or implied by such forward-looking statements.
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About iCo Therapeutics
• Targeting ocular diseases
• Risk-mitigated business model
– Acquire promising drug candidates with identified targets and demonstration of systemic safety
– Develop novel reformulations of drugs
– Expand and create new indications for drugs
• Broad product pipeline
– Focused on underserved markets
– Compelling early data
• Efficient use of capital
• Experienced management & board
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Project Development
iCo Pipeline
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Reformulated Amphotericin B10 indication: Fungal & Parasitic Infections
20 indication: Multiple use delivery platform
Encouraging preclinical/clinical trends10 indication: Wet Age-Related Macular Degeneration
20 indication: Vernal and Atopic Keratoconjunctivitis
Positive safety & efficacy trends10 indication: Diabetic Macular Edema
20 indication: Wet Age-Related Macular Degeneration
Stage
Preclinical
iCo-007
iCo-009
iCo-008Phase 2
Phase 2
iCo holds worldwide exclusive rights to all indications for all projects
iCo–007Second generation
antisense candidate
for the treatment of
DMEPartner: Isis Pharmaceuticals
• Leading cause of blindness in
working age adults
• Proliferation of new blood vessels
which are permeable allows blood to
leak into retinal area, causing
swelling and deformation
• Current treatments:
• Anti VEGF
• Steroids
• Laser
Diabetic Macular Edema
Normal Retina
Abnormal Retina
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Pfizer OSIEyetech
(wAMD & DME)
NovartisGenentech
(wAMD & DME)
BayerRegeneron
(wAMD & DME)
MerckSirna
(wAMD)
Sanofi-AventisFovea
(DME & Other Ophthalmic Therapies)
AlconAstraZeneca
(Ophthalmic Uses for Pipeline)
AlconESBATech
(Ophthalmic Platform)
SantenMacusight
(wAMD & DME)
NovartisAlcon
(Ocular Drug and Device Pipeline)
Validated Therapeutic Arena
7DME = diabetic macular edema
wAMD = wet age-related macular degeneration
U.S. Diabetes
Patients40 million
2006 2025
Leading cause of blindness in
working age adults
1.6 million DME patients today
21million
Assumptions: $10,000 US per
treatment/yr & initial US market
represents only clinically significant cases
Market Opportunity
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10
M
Penetration
500
%
$
Category Product Stage Comments
Device Laser Approved Standard of care for DME
Anti-VEGF
Avastin Off Label No label for DME or wAMD, frequent injections
Macugen Phase 3 Frequent injections, only targets VEGF165 subtype
Lucentis Phase 3Frequent injections, pan VEGF but targets no other pro-
angiogenic factors
VEGF-TRAP Phase 3 „Me too‟ competitor to Lucentis, frequent injections
Steroids
Iluvien, Ozurdex Phase 3 BRVO & CRVO approval for Ozurdex, steroid side effects
Triamcinolone, etc. Off labelSteroid side effects include glaucoma and cataracts
Laser remains gold standard in longitudinal comparison
Competitive Landscape
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iCo-007
• Novel treatment specific for DME
• Mechanism of Action: c-raf kinase-targeted
antisense
• How it improves on current treatments
– Different signaling pathway, multiple targets
– Longer half-life may mean fewer injections
– Easy to manufacture, potential for higher
margins
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Design– Open label
– 15 patients
» 20/63 to 20/500 vision
» OCT at baseline ≥ 250 microns
– Single injection
– Six month follow-up
– Ascending dose ranging
» 110µg, 350µg, 700µg, 1000µg
– Non-responders to previous
treatment
» Steroid, anti-VEGF, laser
Phase 1 Trial Design
Primary EndpointSafety & Tolerability
– Primary end point achieved
– PK below detectable level 2 ng/mL in
blood plasma
Secondary EndpointRetinal Thickness & Visual Acuity
– Encouraging trends warrant further
investigation
– Mean reduction of retinal thickness
169 microns @ 24 wks* (40%
reduction of excess)
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*N=12
KOL‟s and Advisors
KOL’s and AdvisorsDr. Geeta Lalwani
Miami FL
Dr. Scott Cousins
Durham NC
Dr. David Boyer
Beverly Hills CA
Key Phase 1 clinical trial sites
Dr. Victor Gonzalez
McAllen TX
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Dr. David Boyer Dr. Geeta Lalwani
Dr. Scott Cousins Dr. Victor Gonzalez
Dr. Alan Bird
Dr. Philip Rosenfeld
Dr. Jason Slakter
Dr. Karl Csaky
Phase 1 results
Primary Endpoints
No Drug-related SAE‟s
No Signs of Ocular Inflammation
No IOP issues identified in highest doses
No Systemic Exposure
Secondary Endpoints
Mean change in CRT at M6 decrease in CRT at M6 (12/15) = -169 microns
Mean change in reduction of excess RT at W24 (12/15): -40%
All patients who had VA measurement at W24 (13/15) with stable or improved VA compared to baseline (defined as -5 letters or better): 69%
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The number of patients in study is low, statistical significance cannot be inferred
Phase 1: Patient Examples – Single Injection
110ug dose
(sample patient)350ug dose
(sample patient)
528microns
379microns
391microns
228microns
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Delta: 149 microns Delta: 163 microns
Base
line
6 m
onth
s
700ug dose
(sample patient)
867microns
124microns
Delta: 743 microns
1000ug dose
(sample patient)
423microns
187microns
Delta: 236 microns
Phase 2: Randomized, Controlled, Open Label
Multiple iCo-007 injections vs. laser photocoagulation
Primary Endpoints
Change in visual acuity from baseline to month 8 as compared to control group
Secondary Endpoints
Visual acuity & reduction of retinal thickness to month 12
Duration of iCo-007 treatment effect
Safety of repeated iCo-007 injections
Pharmacokinetic (PK) assessments
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Up to 140 patients (5 groups), 12 month follow-up
Doses: 350 & 700 µg (Cohorts 2 & 3 from Phase 1)
iCo-007 Next Steps
√ Received Health Canada Clearance for Phase 2
√ Completed “fill finish” manufacturing program to
produce clinical supply of iCo-007 (February „11)
Phase 2 trial expected to commence ~ early 2011
Discussions with other agencies re: Phase 2
Continue ongoing regional partnership discussions
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iCo–009
1st World: Anti-Fungal
3rd World: Anti-Parasitic
Proprietary oral formulation
of amphotericin B
Partner: The University of British Columbia
iCo-009 Oral Lipid Carrier System
iCo-009
Identify bona fide generics
e.g.
IV Amp B to Oral
Achieve early proof of
relevance
(safety)
Re-start IP Clock
&
Develop Repertoire of
Product Candidates
Anti-fungal, anti-parasitic partnering
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Amphotericin B & IV Delivery
IT WORKS …
– Gold standard
– AmBisome®
• >$400M in sales
• Premium pricing for safety
… BUT NOT PRACTICAL IN MANY SITUATIONS
– Variety of First World fungal infections
– Lack of resources in Developing World
– Inconvenient
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unmet need: oral formulation
Anti-Parasitic Therapeutic Arena
Visceral Leishmaniasis (VL)
• 2nd largest parasitic killer after malaria
• Fatal if left untreated
• 500,000 new infections every year *
• Emerging problem of HIV co-infection
• Symptoms: fever, weight loss, fatigue, anemia and substantial swelling of the liver and spleen
• iCo-009 has Orphan Drug status for VL (September 2010)
* Institute of OneWorld Health
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Proprietary Oral Formulation
Expanding the Amp B market:
Safety and convenience
Oral treatment:Goal of WHO & Gates Foundation
and several other global health
organizations
Non-dilutive funding:CPDD/Gates and CIHR
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Formulation of iCo-009
Lipid capsule – “candy wrapper”
Proprietary platform may be used for other insoluble products, including vaccines and proteins
Preclinical Data
99% Eradication of VL
-BRIEF REPORT, Journal of Infectious Disease 2009,
Kishor M Wasan, et al.
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iCo–008Human monoclonal
antibody treating
both the back and
front of the eye
Partners: AstraZeneca/MedImmune & Immune Pharmaceuticals
iCo-008
• Human monoclonal antibody targeting eotaxin-1
• Good safety & significant clinical history
- Phase 1 & 2 (n=126)
• Highly specific to human eotaxin-1
• Eotaxin binds with high affinity to CCR3
Ocular: Wet Age Related Macular Degeneration (wAMD) &
Vernal & Atopic Keratoconjunctivitis
Systemic: Crohn‟s Disease, IBD, Severe Asthma
(License option granted to Immune Pharmaceuticals)
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US $33M Option with Immune Pharmaceuticals
• iCo retained WW exclusive rights to all ocular applications
• IMPH‟s license option for systemic uses (IBD, severe asthma)
• Non-refundable option fee creditable against a US$1 Million
upfront license fee
• Up to an additional US$32 million in milestone payments
• Royalties on net sales of licensed products
• Immune background: AstraZeneca, Novartis, GSK, Sanofi-
Aventis, Roche, Merck AG, J&J, CV Therapeutics (acquired
by Gilead)
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Wet Age Related Macular Degeneration (wAMD)
• Afflicts the elderly
• Acute event – leads to rapid vision loss
• VEGF validated target
• New literature implicates Eotaxin-1:
– CCR3/Eotaxin-1 axis is a target for age-related macular
degeneration (Nature 2009, Takeda et al.)
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AMD: Preclinical Therapeutic Evidence
Laser-induced CNV in
wild-type mice
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Reduction in CNV
volume for wAMD
50%
eotaxin-1 = CCL11
800
600
400
200
0Ctrl CCL11
Ab
CN
V v
olu
me (
µm
3)x
10
3
Anti-angiogenic
activity for ocular diseases
iCo-008 Clinical History
• Phase 1: (n=25) No serious adverse events
• Phase 2: (n=101) Applied intranasally & topically
– Safe and well tolerated
- Evidence of efficacy in severe allergy indication - reduced post allergy nasal obstruction
Ding et al. Current Opinion in Investigational Drugs 2004 5(11) 28
Upcoming Milestones
• iCo-007
– Phase 2 trial expected to commence ~ early 2011
– Completed “fill finish” manufacturing to produce clinical supply
– Received Health Canada Clearance for Phase 2
– In partnership discussions (regional)
• iCo-009
– Pre-IND meetings have taken place
– Phase 1 trials planned for 2011
– In partnership discussions (regional)
• iCo-008
– Optioned systemic rights to Immune Pharma < US$33 Million
– Retained ocular rights
– Pursuing non-dilutive funding of wAMD program
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Andrew Rae, MBA
Co-founder, Director,
President & CEO
John Clement, PhD
Co-founder, Director & Chief
Technology & Development Officer
Santa Jeremy Ono, PhD
Chief Scientific Officer
Peter Hnik, MD, MHSc.
Chief Medical Officer
John Meekison, BA, CIM, P. Log.
Co-founder & Chief Financial Officer
William Jarosz, JD
Chairman of the Board, iCo
Cartesian Capital Group, LLC
Richard Barker, PhD
Director General of the
Association of the British
Pharmaceutical Industry
Noel Hall
Co-founder of Aspreva
Donald Buell, MD
iCo-009 SAB Chair, Former Senior
Medical Director, Astellas USA
George Lasezkay, JD
Principal, Turning Point Consultants,
LLC, Former VP Corp Development,
Allergan
Non-Executive Directors Strategic Advisory Board
Extensive public company and life science experience | Solid operational and product
development expertise | Ophthalmic specific expertise
Management and Directors
Management
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$10 Million Equity Line Facility (ELF)
• Dutchess Opportunity Cayman Fund Limited ("Dutchess") committed to
provide up to $10M in equity capital over the next 3 years
• Newly issued common shares subject to a minimum price set by iCo
• No commission, warrants or any other derivative
• iCo may chose to draw on the ELF at iCo´s sole discretion
• iCo must file and clear a short-form shelf prospectus with the
applicable securities authorities in Canada
Flexible source of capital at reasonable terms
iCo can remain opportunistic re: ongoing corporate finance & strategic
activities
Financials (As of Year End: December 31, 2010)
Filings available at www.sedar.com
Invested Capital to Date$18.7 million
Isis Pharmaceuticals largest shareholder (12.3%)
Cash $2 million
Burn Approx $0.6 million/qtr
Share Capital
41.1 M Shares Outstanding
42.9 M Fully Diluted
1.9 M Options
Head Office Vancouver BC, Canada
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Summary
• Targeting ocular diseases
• Risk-mitigated business model
– Acquire promising drug candidates with identified targets and demonstration of systemic safety
– Develop novel reformulations of drugs
– Expand and create new indications for drugs
• Broad product pipeline
– Focused on underserved markets
– Compelling early data
• Efficient use of capital
• Experienced management & board
33
iCo TherapeuticsTSX-V: ICO
604-602-9414
www.icotherapeutics.com